Geron Presents Data from GRNOPC1 Trial at International Conferences on Spinal
Cord Medicine and Rehabilitation
MENLO PARK, Calif., Jun 07, 2011 (BUSINESS WIRE) -- Geron Corporation (GERN)
today announced two presentations on the company's ongoing Phase 1 clinical trial
of the human embryonic stem cell-based therapy, GRNOPC1, in patients with spinal
cord injury. Data on the first two patients were presented at the 2011
International Conference on Spinal Cord Medicine and Rehabilitation in
Washington, D.C. A second presentation was given at the 2011 Spine Symposium,
which was held as part of The American Spinal Injury Association (ASIA) Annual
Scientific Meeting. The presentations were given by Edward Wirth, III, M.D.,
Ph.D., Geron's Medical Director for Cell Therapies and Linda Jones, P.T., M.S.,
Geron's Senior Clinical Trials Manager for GRNOPC1.
"We are pleased to report a very good safety profile of GRNOPC1 to date, with no
serious adverse events," said Dr. Wirth. "Currently, we have two patients in the
trial. We anticipate increased enrollment going forward as we open our additional
clinical sites and following FDA approval to expand our inclusion criteria to
patients with injuries resulting in a neurological level of T11, which represents
a substantial percentage of thoracic injuries, and to reduce the stagger between
patients from 30 days to 10 days."
Phase 1 Clinical Trial Data
Data were presented on two patients with neurologically complete American Spinal
Injury Association (ASIA) Impairment Scale grade A thoracic spinal cord injuries,
who received GRNOPC1 at a dose of 2 million cells delivered by injection into the
lesion site using a syringe positioning device specially designed by Geron.
GRNOPC1 was administered between 7 and 14 days after injury. Low-dose tacrolimus
was given for temporary immune-suppression from the time of injection for 46
days, at which point the dose was tapered and withdrawn completely at 60 days.
Endpoints of the trial are safety and neurological function, using standardized
testing at specified timepoints to monitor sensory and lower extremity motor
function. The trial protocol also includes multiple MRI scans. One patient in the
trial has completed the Day 180 follow-up visit and the other has completed Day 7
follow-up.
There were no surgical complications during or after either procedure. No adverse
events have occurred to date related to the injection procedures or to GRNOPC1.
One patient experienced two mild adverse events related to tacrolimus, nausea and
low magnesium, which are known to be associated with the immune-suppressive drug
and were minor. There is no evidence of cavitation in the spinal cord at the
injury site on MRI through Day 180. Initial analyses have shown no evidence of
immune responses to GRNOPC1 through Day 90, which includes 30 days after complete
withdrawal of immune-suppression.
Regulatory & Operational Update
The company has received clearance from the FDA to expand eligibility criteria
from injuries between spinal segments T3 and T10 to include patients with
injuries at T11. In addition, the FDA has approved that the current 30 day period
between subjects in the trial may be reduced to 10 days.
Five clinical sites are currently open for patient enrollment, with two
additional sites to follow. All sites are referral medical centers for spinal
cord trauma and/or rehabilitation, and the catchment area for the clinical trial
now includes all 48 lower U.S. States.
GRNOPC1 Clinical Program
In this first cohort of patients, a dose of 2 million cells is being administered
to assess safety in up to 10 patients with neurologically complete injuries in
the thoracic region of the spinal cord. Pending demonstration of safety in that
patient population, clinical testing will be expanded to patients with injuries
to the cervical region and to patients with neurologically incomplete injuries,
as well as to assess escalating doses of GRNOPC1.
Program Funding
In May, Geron announced a Targeted Clinical Development Award totaling $25
million from the California Institute for Regenerative Medicine (CIRM) to support
the clinical development of GRNOPC1. The Award will support funding of clinical
trial costs, related non-clinical studies, analytical development and the
manufacture of cells to be used in the clinical trial. CIRM funding will provide
matching support as Geron progresses the Phase 1 clinical program in spinal cord
injury.
About GRNOPC1
GRNOPC1 contains hESC-derived oligodendrocyte progenitor cells that have
demonstrated remyelinating, nerve growth stimulating and angiogenic properties
leading to restoration of function in animal models of acute spinal cord injury.
Preclinical studies have shown that administration of GRNOPC1 significantly
improved locomotor activity and kinematic scores of animals with spinal cord
injuries when injected seven days after the injury. Histological examination of
the injured spinal cords treated with GRNOPC1 showed improved axon survival and
extensive remyelination surrounding the rat axons. A Phase 1 clinical trial of
GRNOPC1 has been initiated in patients with complete ASIA Impairment Scale grade
A thoracic spinal cord injuries. For more information about GRNOPC1, visit
geron.com. For further information about the Phase 1
clinical trial, including location of clinical sites, visit
clinicaltrials.gov.
About Spinal Cord Injury
Spinal cord injury is caused by trauma to the spinal cord that results in a loss
of motor control, sensatory perception or bowel and bladder control. A traumatic
blow to the spine can fracture or dislocate vertebrae that may cause bone
fragments or disc material to injure the nerve fibers and damage the
oligodendrocyte cells that insulate the nerve fibers in the spinal cord. Most
human spinal cord injuries are contusions (bruises) to the cord, rather than a
severance of the nerve fibers. Every year approximately 12,000 people in the U.S.
sustain spinal cord injuries. There are currently no approved therapies for the
treatment of spinal cord injury.
About Geron
Geron is developing first-in-class biopharmaceuticals for the treatment of cancer
and chronic degenerative diseases. The company is advancing anti-cancer therapies
through multiple Phase 2 clinical trials in different cancers by targeting the
enzyme telomerase and with a compound designed to penetrate the blood-brain
barrier. The company is developing cell therapies from differentiated human
embryonic stem cells for a range of indications, with the first product in a
Phase 1 clinical trial for spinal cord injury. For more information, visit
geron.com. |
